A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision loss

Phase 1

• Conditions: Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene; MedDRA version: 20.0Level: PTClassification code 10038914Term: Retinitis pigmentosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2021-002729-74-DK
• Lead Sponsor: ProQR Therapeutics IV B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-11-26
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 81

Inclusion Criteria

- An adult (= 18 years) willing and able to provide informed consent for participation prior to performing any study related procedure. OR A minor able to complete all study assessments and comply with the protocol and has a parent or caregiver willing and able to follow study instructions, and attend study visits with the subject as required.
- Clinical presentation consistent with RP with Usher syndrome type 2 or non-syndromic form of RP (NSRP), based on ophthalmic, audiologic, and vestibular examinations.
- A molecular diagnosis of homozygosity or compound heterozygosity for 1 or more pathogenic exon 13 mutations in the USH2A gene, based on genetic analysis at screening.
- Reliable BCVA, perimetry, and other measurements in both eyes.

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 74
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

- Presence of any significant ocular or non-ocular disease/disorder (or medication and/or laboratory test abnormalities) which, in the opinion of the Investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or the subject’s ability to participate in the study.
- Known hypersensitivity to antisense oligonucleotides or any constituents of the injection.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified